This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
322
Stage A: efgartigimod PH20 SC, Stage B: efgartigimod PH20 SC
Stage A: N/A, stage B: placebo
Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI)
Time frame: Up to 12 weeks during the open-label stage A
Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score
The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score
The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
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Investigator site 0010065
Birmingham, Alabama, United States
Investigator site 0010013
Phoenix, Arizona, United States
Investigator site 0010055
Scottsdale, Arizona, United States
Investigator Site 0010032
Carlsbad, California, United States
Investigator site 0010004
Orange, California, United States
Investigator site 0010190
Pomona, California, United States
Investigator site 0010160
Rancho Mirage, California, United States
Investigator site 0010071
San Francisco, California, United States
Investigator site 0010057
Centennial, Colorado, United States
Investigator site 0010026
New Haven, Connecticut, United States
...and 206 more locations
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength
This is measured with a handheld device called a vigometer
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Time frame: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Time frame: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))
Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
Time frame: Up to 12 weeks during the open-label stage A
Stage A: Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time
Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.
Time frame: Up to 12 weeks during the open-label stage A
Stage B: Time to CIDP Disease Progression
Time to chronic inflammatory demyelinating polyneuropathy (CIDP) disease progression is defined by the time from first dose of double-blind IMP to the first I-RODS score decrease ≥4 points compared to Stage B baseline using the centile metric.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Number of Participants With Improved Functional Level Compared to Stage B Baseline
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Adjusted INCAT Score
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in MRC Sum Score
The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in 24-item I-RODS Disability Score
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in TUG Score
The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Mean Grip Strength
This is measured with a handheld device called a vigometer
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Time to 10% Decrease in the 24-item I-RODS
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Percent Changes of Serum IgG Levels Over Time
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Changes From Stage B Baseline to Last Assessment in Stage B, in EQ-5D-5L Visual Analog Scale (VAS) Over Time
Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.
Time frame: Up to 48 weeks during the randomized placebo-controlled stage B